Carolyn Wang

I love the Dr. Peter Marks @US_FDA truth bombs, and there were many today at #NARDS. We're closing on a good one: "Genomic medicine is here to stay. It's about [what country] will get the components together first to make it possible." (here with @Penn ODC Dir. James Wilson, MD)

#PMWC23: Peter Marks @us_fda: "We should not insist on mid-20th trial design." #raredisease @ultragenyx

#PMWC23: Peter Marks @us_fda talks #biomarkers in with @ultragenyx CEO Emil Kakkis & others: "In rare diseases with limited natural history data, we need to think about using self controls and biomarkers that show [change] over the intervention period."

Closing out #AorticDissectionAwarenessWeek, I encourage everyone to make a #purplepinkypromise to learn more about your aortic health. Go to bit.ly/3RWQFaU for your Aorta IQ and ask friends and loved ones to make the promise! @JohnRitterFdn

Emil Kakkis @ultragenyx spoke @ResearchAmerica today on current limitations of drug dev process for #raredisease: If you measure a disease by clinical symptoms, it may be too late. "Biomarkers are not a compromise, they are the right way to support development of these drugs."

Dr. Emil Kakkis @MilkenInstitute @fastercures panel on healthcare access: We committed to ensuring anyone in the US that can benefit from our therapies can access them. It's harder ex-US but we committed to majority access and trying to fill gaps where we can. #MLGlobal $RARE

.@JayasreeKIyer on @MilkenInstitute panel: Most treatments reach about 10% of those they could benefit. We cannot wait for US and EU approvals to get therapies registered in other parts of the world. #MIGlobal

With Dr. Emil Kakkis of $RARE @MilkenInstitute Global Conference where he's sharing his family story and personal path in #raredisease for the Milken Center Advancing the American Dream project. #MIGlobal

Jessica Mega @verilylifesci speaks with @CliftonLeaf @rajpanjabi @drhew at @FortuneMagazine Brainstorm Health: rolling out testing was not unlike launching a clinical study: how do you test those who need it most, schedule people at the right sites, and capture the right data

@vivianleemd: "Universities and employers are asking how do we know that we can bring people back? What test should I be using? By layering on data science and epidemiologic models we can help guide these decisions." @nytimes w/ @natashanyt @vivek_murthy @verilylifesci

It's International Overdose Awareness Day - People in need who are in Dayton, Ohio can access treatment immediately at 937-535-5115. onefifteen.org #endoverdose @verilylifesci